CA2576923A1 - Vecteur - Google Patents

Vecteur Download PDF

Info

Publication number
CA2576923A1
CA2576923A1 CA002576923A CA2576923A CA2576923A1 CA 2576923 A1 CA2576923 A1 CA 2576923A1 CA 002576923 A CA002576923 A CA 002576923A CA 2576923 A CA2576923 A CA 2576923A CA 2576923 A1 CA2576923 A1 CA 2576923A1
Authority
CA
Canada
Prior art keywords
liposome
sirna
delivery vector
vector according
group
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Abandoned
Application number
CA002576923A
Other languages
English (en)
Inventor
Michael Keller
Michael Jorgensen
Andrew David Miller
Eric Perouzel
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
IC Vec Ltd
Original Assignee
Individual
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Individual filed Critical Individual
Publication of CA2576923A1 publication Critical patent/CA2576923A1/fr
Abandoned legal-status Critical Current

Links

Classifications

    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00Medicinal preparations characterised by special physical form
    • A61K9/10Dispersions; Emulsions
    • A61K9/127Liposomes
    • A61K9/1271Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers
    • A61K9/1272Non-conventional liposomes, e.g. PEGylated liposomes, liposomes coated with polymers with substantial amounts of non-phosphatidyl, i.e. non-acylglycerophosphate, surfactants as bilayer-forming substances, e.g. cationic lipids
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0091Purification or manufacturing processes for gene therapy compositions
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61PSPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/87Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation
    • C12N15/88Introduction of foreign genetic material using processes not otherwise provided for, e.g. co-transformation using microencapsulation, e.g. using amphiphile liposome vesicle
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K2121/00Preparations for use in therapy
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • YGENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10TTECHNICAL SUBJECTS COVERED BY FORMER US CLASSIFICATION
    • Y10T428/00Stock material or miscellaneous articles
    • Y10T428/29Coated or structually defined flake, particle, cell, strand, strand portion, rod, filament, macroscopic fiber or mass thereof
    • Y10T428/2982Particulate matter [e.g., sphere, flake, etc.]
    • Y10T428/2984Microcapsule with fluid core [includes liposome]

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Genetics & Genomics (AREA)
  • Engineering & Computer Science (AREA)
  • Chemical & Material Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Biotechnology (AREA)
  • Molecular Biology (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Organic Chemistry (AREA)
  • Animal Behavior & Ethology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Wood Science & Technology (AREA)
  • Biomedical Technology (AREA)
  • Epidemiology (AREA)
  • Biophysics (AREA)
  • Dispersion Chemistry (AREA)
  • Physics & Mathematics (AREA)
  • Manufacturing & Machinery (AREA)
  • Plant Pathology (AREA)
  • Microbiology (AREA)
  • Biochemistry (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Medicinal Preparation (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

La présente invention concerne un vecteur de livraison non viral comprenant un liposome, dans lequel un ou plusieurs lipides du liposome sont couplés, d~une manière réversible ou irréversible, à un ou plusieurs polymères et dans lequel le liposome comprend du siARN.
CA002576923A 2004-08-13 2005-07-06 Vecteur Abandoned CA2576923A1 (fr)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
GB0418172.3 2004-08-13
GBGB0418172.3A GB0418172D0 (en) 2004-08-13 2004-08-13 Vector
US61408504P 2004-09-09 2004-09-09
US60/614,085 2004-09-09
PCT/GB2005/002634 WO2006016097A2 (fr) 2004-08-13 2005-07-06 Vecteur

Publications (1)

Publication Number Publication Date
CA2576923A1 true CA2576923A1 (fr) 2006-02-16

Family

ID=33017523

Family Applications (1)

Application Number Title Priority Date Filing Date
CA002576923A Abandoned CA2576923A1 (fr) 2004-08-13 2005-07-06 Vecteur

Country Status (8)

Country Link
US (1) US20080063701A1 (fr)
EP (1) EP1814594A2 (fr)
CN (1) CN101094691A (fr)
AU (1) AU2005271100A1 (fr)
CA (1) CA2576923A1 (fr)
GB (1) GB0418172D0 (fr)
WO (1) WO2006016097A2 (fr)
ZA (1) ZA200701246B (fr)

Families Citing this family (30)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
BRPI0713331A2 (pt) * 2006-06-30 2012-03-13 Hokkaido System Science Co., Ltd. composição para a transfecção de ácido nucléico
EP2125031B1 (fr) 2006-12-19 2017-11-01 Marina Biotech, Inc. Lipides et ensembles lipidiques comprenant des éléments d'amélioration de la transfection
AR067997A1 (es) * 2007-08-24 2009-10-28 Novartis Ag Compuestos organicos
GB2458473A (en) 2008-03-17 2009-09-23 Imuthes Ltd 3'-O-allyl- and 3'-O-carboxymethyl- 2'-aminosaccharide derivatives, & amides thereof with peptides, as adjuvants
WO2009124137A2 (fr) * 2008-04-01 2009-10-08 Mount Sinai School Of Medicine Of New York University Procédé de suppression d’une transcription de gène par méthylation d’histone lysine
CN102112110A (zh) * 2008-06-06 2011-06-29 米尔纳医疗股份有限公司 用于RNAi试剂体内递送的新型组合物
EP2184054A1 (fr) 2008-11-08 2010-05-12 Lipoxen Technologies Limited Livraison ARN à faible interférence
AU2010210593B2 (en) * 2009-02-04 2015-09-03 The Brigham And Women's Hospital, Inc. Nanoscale platinum compounds and methods of use thereof
PT3338765T (pt) 2009-12-01 2019-03-18 Translate Bio Inc Derivado de esteróide adequado para a administração de arnm em doenças genéticas humanas
SG10201407996PA (en) 2009-12-23 2015-01-29 Novartis Ag Lipids, lipid compositions, and methods of using them
US9028797B2 (en) 2010-02-26 2015-05-12 Nagasaki University Composite body for antigen or drug delivery
AU2012267531B2 (en) 2011-06-08 2017-06-22 Translate Bio, Inc. Lipid nanoparticle compositions and methods for mRNA delivery
US20140161876A1 (en) * 2011-07-15 2014-06-12 Konica Minolta, Inc. Liposome-containing preparation utilizing dissolution aid, and method for producing same
CN102973506B (zh) * 2011-09-05 2015-06-03 中国科学院深圳先进技术研究院 阳离子脂质体及其制备方法
GB201116248D0 (en) * 2011-09-20 2011-11-02 Glaxosmithkline Biolog Sa Liposome production using isopropanol
EP3536787A1 (fr) 2012-06-08 2019-09-11 Translate Bio, Inc. Polynucléotides résistant aux nucléases et leurs utilisations
EA201492055A1 (ru) 2012-06-08 2015-11-30 Шир Хьюман Дженетик Терапис, Инк. ИНГАЛЯЦИОННАЯ ДОСТАВКА мРНК В НЕЛЕГОЧНЫЕ КЛЕТКИ-МИШЕНИ
JP6586075B2 (ja) 2013-03-14 2019-10-02 トランスレイト バイオ, インコーポレイテッド メッセンジャーrnaの精製方法
HUE042640T2 (hu) 2013-03-14 2019-07-29 Translate Bio Inc CFTR-mRNS-készítmények, valamint kapcsolódó eljárások és alkalmazások
EP3060258A1 (fr) 2013-10-22 2016-08-31 Shire Human Genetic Therapies, Inc. Thérapie à l'arnm pour la phénylcétonurie
ES2707966T3 (es) 2013-10-22 2019-04-08 Translate Bio Inc Terapia de ARNm para la deficiencia en síntesis de argininosuccinato
KR20220158867A (ko) 2014-04-25 2022-12-01 샤이어 휴먼 지네틱 테라피즈 인크. 메신저 rna 의 정제 방법
EP3169309B1 (fr) 2014-07-16 2023-05-10 Novartis AG Procédé d'encapsulation d'un acide nucléique dans une nanoparticule lipidique hôte
CN105985386B (zh) 2015-02-11 2018-06-26 大连民族学院 一类蔗糖酯型阳离子基因载体及其制备方法
CZ307452B6 (cs) * 2016-11-03 2018-09-05 Vysoká škola chemicko-technologická v Praze Aminooxylipidy pro konstrukci samoskladných liposomálních systémů umožňujících jejich následnou modifikaci biologicky funkčními molekulami
EP3585417B1 (fr) 2017-02-27 2023-02-22 Translate Bio, Inc. Méthode de préparation d'arnm cftr à codons optimisés
WO2018213476A1 (fr) 2017-05-16 2018-11-22 Translate Bio, Inc. Traitement de la fibrose kystique par administration d'arnm à codons optimisés codant pour la cftr
CA3108544A1 (fr) 2018-08-24 2020-02-27 Translate Bio, Inc. Procedes de purification d'arn messager
CN113116819B (zh) * 2021-04-24 2022-09-20 郑州大学 一种载siRNA纳米脂质杂化胶束及其制备方法和应用
CN114249791A (zh) * 2021-12-27 2022-03-29 北京工商大学 一种甾醇衍生的酰胺基寡肽型表面活性剂及其制备方法

Family Cites Families (11)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
AU2911197A (en) * 1996-05-24 1998-01-05 Imperial College Of Science, Technology And Medicine Polycationic sterol derivatives as transfection agents
US6120751A (en) * 1997-03-21 2000-09-19 Imarx Pharmaceutical Corp. Charged lipids and uses for the same
GB9930533D0 (en) * 1999-12-23 2000-02-16 Mitsubishi Tokyo Pharm Inc Nucleic acid delivery
US20020188023A1 (en) * 2000-12-12 2002-12-12 Michael Jorgensen Compound
US6921812B1 (en) * 2001-07-03 2005-07-26 Isis Pharmaceuticals, Inc. Methods of modulating pharmacokinetics of oligonucleotides
AU2003237249A1 (en) * 2002-05-24 2003-12-12 Isis Pharmaceuticals, Inc. Oligonucleotides having modified nucleoside units
US7148342B2 (en) * 2002-07-24 2006-12-12 The Trustees Of The University Of Pennyslvania Compositions and methods for sirna inhibition of angiogenesis
US20050002999A1 (en) * 2003-06-04 2005-01-06 Rahul Mehta Long-circulating liposomal compositions
EP2567693B1 (fr) * 2003-07-16 2015-10-21 Protiva Biotherapeutics Inc. ARN interférents encapsulés dans des lipides
JP4842821B2 (ja) * 2003-09-15 2011-12-21 プロチバ バイオセラピューティクス インコーポレイティッド ポリエチレングリコール修飾脂質化合物およびその使用
WO2005121348A1 (fr) * 2004-06-07 2005-12-22 Protiva Biotherapeutics, Inc. Arn interferant encapsule dans des lipides

Also Published As

Publication number Publication date
ZA200701246B (en) 2008-12-31
GB0418172D0 (en) 2004-09-15
AU2005271100A1 (en) 2006-02-16
US20080063701A1 (en) 2008-03-13
WO2006016097A2 (fr) 2006-02-16
CN101094691A (zh) 2007-12-26
WO2006016097A3 (fr) 2006-11-23
EP1814594A2 (fr) 2007-08-08

Similar Documents

Publication Publication Date Title
US20080063701A1 (en) Vector
Zhen et al. Liposomal delivery of CRISPR/Cas9
Sun et al. Structure and function of cationic and ionizable lipids for nucleic acid delivery
Wu et al. Lipidic systems for in vivo siRNA delivery
Kapoor et al. Physicochemical characterization techniques for lipid based delivery systems for siRNA
Romberg et al. Sheddable coatings for long-circulating nanoparticles
Rao Cationic lipid-mediated nucleic acid delivery: beyond being cationic
EP1244805B1 (fr) Complexes d'administration a base d'acides nucleiques-lipide cationique-proteine noyau virale
US20150374842A1 (en) Process for formulating an anionic agent
Mokhtarieh et al. Asymmetric liposome particles with highly efficient encapsulation of siRNA and without nonspecific cell penetration suitable for target-specific delivery
EP3673898A1 (fr) Procédé de production de nanoparticules lipidiques pour l'administration de médicaments
JP5801055B2 (ja) 標的遺伝子の発現を抑制する組成物
US20100297023A1 (en) Lipid
Uddin Cationic lipids used in non-viral gene delivery systems
Yan et al. Lipid nanovehicles overcome barriers to systemic RNA delivery: lipid components, fabrication methods, and rational design
Bartsch et al. Cell-specific targeting of lipid-based carriers for ODN and DNA
JP2008509205A (ja) ポリマー修飾siRNAリポソームを含むベクター
US11793756B2 (en) Anionic nanocomplexes for nucleic acid delivery
Eş et al. Engineering aspects of lipid-based delivery systems: In vivo gene delivery, safety criteria, and translation strategies
KR101647178B1 (ko) 효소 절단성 링커 또는 올리고 라이신을 포함하는 폴리에틸렌글리콜-리피드를 이용한 안정화된 플라스미드-지질 입자
Kostarelos et al. What role can chemistry play in cationic liposome‐based gene therapy research today?
Martin et al. Advances in cationic lipid-mediated gene delivery
Tavano et al. Nanovesicular formulations for cancer gene therapy
JP5872898B2 (ja) 標的遺伝子の発現を抑制する組成物
JP2007166946A (ja) 標的遺伝子の発現を抑制するための組成物

Legal Events

Date Code Title Description
FZDE Discontinued
FZDE Discontinued

Effective date: 20110706